Ultresa

"The U.S. Food and Drug Administration yesterday approved Ruconest, the first recombinant C1-Esterase Inhibitor product for the treatment of acute attacks in adult and adolescent patients with hereditary angioedema (HAE).

Ultresa

Drug Description

Side Effects

Interactions

SIDE EFFECTS

The most serious adverse reactions reported with
different pancreatic enzyme products of the same active ingredient
(pancrelipase) that are described elsewhere in the label include fibrosing
colonopathy, hyperuricemia and allergic reactions [see WARNINGS AND
PRECAUTIONS].

Clinical Trials Experience

Because clinical trials are conducted under widely
varying conditions, adverse reaction rates observed in the clinical trials of a
drug cannot be directly compared to rates in the clinical trials of another
drug and may not reflect the rates observed in clinical practice.

The short-term safety of ULTRESA was assessed in two
clinical trials conducted in 40 patients with exocrinepancreatic insufficiency
(EPI) due to cystic fibrosis (CF). Study 1 was conducted in 31 patients, ages 8
years to 37 years; Study 2 was conducted in 9 patients, ages 7 years to 11
years.

Study 1 was a randomized, double-blind, placebo-controlled,
crossover study of 31 patients, ages 8 to 37 years, with EPI due to CF. In this
study, patients were randomized to receive ULTRESA at doses not to exceed 2,500
lipase units per kilogram per meal or matching placebo for 6 to 7 days of treatment,
followed by crossover to the alternate treatment for an additional 6 to 7 days.
The mean daily dose of ULTRESA was 6,270 lipase units per kilogram body weight
per day. The mean exposure to ULTRESA during this study was 5.4 days.

The most common adverse reactions ( ≥ 7%) were
headache, pharyngolaryngeal pain, and epistaxis. Table 1 enumerates adverse
reactions that occurred in at least 2 patients (greater than or equal to 7%)
treated with ULTRESA at a higher rate than with placebo in Study 1.

Study 2 was an open-label study of 9 patients, ages 7
years to 11 years, with EPI due to CF. After a screening period of up to 15
days on individually-titrated doses of ULTRESA not to exceed 2,500 lipase units
per kilogram per meal, patients entered a washout phase (no treatment) of up to
7 days before returning to a treatment phase of up to 12 days on the same
individually-titrated dose of ULTRESA. Two patients discontinued during the
washout phase leaving 7 patients in the treatment phase. The mean daily dose of
ULTRESA was 6,361 lipase units per kilogram body weight per day during the last
4 days of the screening phase, and was 6,846 lipase units per kilogram body
weight per day during the treatment phase. The mean duration of the treatment
phase was 5.7 days.

Postmarketing Experience

Postmarketing data for ULTRESA has been available since
2003. The safety data is similar to that described below. Because these
reactions are reported voluntarily from a population of uncertain size, it is
not always possible to reliably estimate their frequency or establish a causal
relationship to drug exposure.

Pancreatic enzyme products (delayed and
immediate-release) with different formulations of the same active ingredient
(pancrelipase) have been used for the treatment of patients with exocrine
pancreatic insufficiency due to cystic fibrosis and other conditions, such as
chronic pancreatitis. The long-term safety profile of these products has been
described in the medical literature. The most serious adverse events included
fibrosing colonopathy, distalintestinal obstruction syndrome (DIOS),
recurrence of pre-existing carcinoma, and severe allergic reactions including
anaphylaxis, asthma, hives, and pruritus. The most commonly reported adverse
events were gastrointestinal disorders, including abdominal pain, diarrhea,
flatulence, constipation and nausea, and skin disorders including pruritus,
urticaria and rash.